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PubMed Journals Articles About "Therachon Receives Orphan Drug Designation Achondroplasia" RSS

03:57 EDT 24th September 2018 | BioPortfolio

Therachon Receives Orphan Drug Designation Achondroplasia PubMed articles on BioPortfolio. Our PubMed references draw on over 21 million records from the medical literature. Here you can see the latest Therachon Receives Orphan Drug Designation Achondroplasia articles that have been published worldwide.

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Showing "Therachon receives orphan drug designation achondroplasia" PubMed Articles 1–25 of 9,900+

Orphan drugs: trends and issues in drug development.

Research in rare diseases has contributed substantially toward the current understanding in the pathophysiology of the common diseases. However, medical needs of patients with rare diseases have always been neglected by the society and pharmaceutical industries based on their small numbers and unprofitability. The Orphan Drug Act (1983) was the first serious attempt to address the unmet medical needs for patients with rare diseases and to provide impetus for the pharmaceutical industry to promote orphan dru...


Acute type A aortic dissection repair in an octogenarian with achondroplasia: a case report.

Achondroplasia is an inherited disorder and the most common type of short-limbed dwarfism in human beings, affecting more than 250,000 individuals worldwide. To the best of our knowledge, no study has reported a correlation between achondroplasia and aortic dissection. Here, we report a rare case of acute type A aortic dissection repair in a patient with achondroplasia.

Growth velocity and biological variables during puberty in achondroplasia.

Achondroplasia is the most common form of inherited disproportionate short stature. Cross-sectional design studies of height show that, during childhood, height standard deviation scores (SDS) declines steadily and reaches a mean adult height at -6.42 and -6.72 SDS. However, there is a lack of knowledge about longitudinal growth and biological variables during puberty for children with achondroplasia. Here we report the growth velocity and biological parameters during puberty in children with achondroplasia...


Comparative oncology approach to drug repurposing in osteosarcoma.

Osteosarcoma is an orphan disease for which little improvement in survival has been made since the late 1980s. New drug discovery for orphan diseases is limited by the cost and time it takes to develop new drugs. Repurposing already approved FDA-drugs can help overcome this limitation. Another limitation of cancer drug discovery is the lack of preclinical models that accurately recapitulate what occurs in humans. For OS using dogs as a model can minimize this limitation as OS in canines develops spontaneous...

Early postnatal soluble FGFR3 therapy prevents the atypical development of obesity in achondroplasia.

Achondroplasia is a rare genetic disease is characterized by abnormal bone development and early obesity. While the bone aspect of the disease has been thoroughly studied, early obesity affecting approximately 50% of them during childhood has been somewhat neglected. It nevertheless represents a major health problem in these patients, and is associated to life-threatening complications including increasing risk of cardiovascular pathologies. We have thus decided to study obesity in patients and to use the m...

The FDA Breakthrough-Drug Designation - Four Years of Experience.

FDA Expectations for Demonstrating Interchangeability.

There is a great interest from global companies who are developing biosimilars to pursue interchangeability designation for commercialization of their products in the US. An interchangeability designation will not only allow the substitutability at the pharmacy level without the intervention of a health care provider but the first sponsor who is able to garner interchangeability designation will also receive 12 months of marketing exclusivity. This paper will highlight our current understanding of FDA expec...

Growth in achondroplasia: Development of height, weight, head circumference, and body mass index in a European cohort.

As growth references for achondroplasia are limited to reports from United States, Japan, Argentina, and Australia, the aim of this study was to construct growth references for height, weight, head circumference, and body mass index (BMI) from a European cohort of children with achondroplasia and to discuss the development of these anthropometric variables. A mix of cross-sectional and longitudinal, retrospective, and prospective data from 466 children with achondroplasia and 4,375 measuring occasions were ...

Dose-Finding Studies Among Orphan Drugs Approved in the EU: A Retrospective Analysis.

In the development process for new drugs, dose-finding studies are of major importance. Absence of these studies may lead to failed phase 3 trials and delayed marketing authorization. In our study we investigated to what extent dose-finding studies are performed in the case of orphan drugs for metabolic and oncologic indications. We identified all orphan drugs that were authorized until August 1, 2017. European Public Assessment Reports were used to extract the final dose used in the summary of product char...

Polysomnography as an indicator for cervicomedullary decompression to treat foramen magnum stenosis in achondroplasia.

Management of cervicomedullary compression due to foramen magnum stenosis in achondroplasia remains controversial, especially for patients with no symptoms or mild symptoms. We examined the effectiveness of polysomnography (PSG) as an indicator for cervicomedullary decompression treatment.

Validation of the binary designation 'Symbiodinium thermophilum' (Dinophyceae).

The binary designation 'Symbiodinium thermophilum' was invalid due to the absence of an illustration as required by Article 44.2 of the ICN. Herein, it is validated. This species is the most common symbiont in reef corals in the southern Persian/Arabian Gulf (PAG), the world's hottest body of water sustaining reef coral growth. This article is protected by copyright. All rights reserved.

Total Joint Arthroplasty Quality Ratings: How Are They Similar and How Are They Different?

A patient's perception of hospital or provider quality can have far-reaching effects, as it can impact reimbursement, patient selection of a surgeon, and healthcare competition. A variety of organizations offer quality designations for orthopedic surgery and its subspecialties. Our goal is to compare total joint arthroplasty (TJA) quality designation methodology across key quality rating organizations. One researcher conducted an initial Google search to determine organizations providing quality designation...

Urrets-Zavalia syndrome post keratoplasty, keratoconus and achondroplasia: Is there a link?

Changes in the range of motion of the lower limb joints during extensive tibial lengthening in achondroplasia.

Increase in the magnitude of lengthening during the limb lengthening procedure involves a high risk of decreased range of motion (ROM) in adjacent joints. Even though patients with achondroplasia can tolerate a relatively larger amount of lengthening owing to its inherent soft-tissue laxity, they often exhibit significant joint contractures during extensive lengthening. In the present study, we evaluated temporal changes in the ROM of the hip, knee, and ankle joints throughout the treatment period in 12 lim...

Magnetic work environments: Patient experience outcomes in Magnet versus non-Magnet hospitals.

The term Magnet hospital is an official designation ascribed by the American Nurses Credentialing Center for hospitals that meet specific criteria indicating they have a "magnetic work environment" for nurses. The objective of the Magnet designation is to encourage hospitals to design work in such a way as to attract and retain high-quality nurses and thus improve the quality of patient care. Empirical research has demonstrated that hospitals who earn a Magnet designation appear to have nurses who are more ...

Specialty Drug Coverage Varies Across Commercial Health Plans In The US.

We analyzed specialty drug coverage decisions issued by the largest US commercial health plans to examine variation in coverage and the consistency of those decisions with indications approved by the Food and Drug Administration (FDA). Across 3,417 decisions, 16 percent of the 302 drug-indication pairs were covered the same way by all of the health plans, and 48 percent were covered the same way by 75 percent of the plans. Specifically, 52 percent of the decisions were consistent with the FDA label, 9 ...

Psychological wellbeing of children at public primary schools in Jimma town: An orphan and non-orphan comparative study.

Orphans face multiple challenges including insufficient food, shelter, schooling, and medical care. Most research on orphans in developing countries concentrates on nutrition and health status. The present study aims to explore the psychological wellbeing of in-school orphaned and non-orphaned children.

Severe poisoning with naphazoline: update from a therapeutic error.

Naphazoline is a drug commonly used as a decongestant in adult patients. Its indication in Pediatrics is not frequent, being approved its use from the age of 12 for the toxic effects it possesses. Intoxication in children generates a potentially serious clinical picture. It is characterized by the immediate appearance of hypotonia, deterioration of the sensory, hypothermia and bradycardia of variable degree of clinical compromise. Although it is an infrequent intoxication, the anamnesis and the initial mana...

The Orphan Receptor GPR35 Contributes to Angiotensin II-Induced Hypertension and Cardiac Dysfunction in Mice.

The orphan receptor G protein-coupled receptor 35 (GPR35) has been associated with a range of diseases, including cancer, inflammatory bowel disease, diabetes, hypertension, and heart failure. To assess the potential for GPR35 as a therapeutic target in cardiovascular disease, this study investigated the cardiovascular phenotype of a GPR35 knockout mouse under both basal conditions and following pathophysiological stimulation.

Breakthrough-Therapy Designation - An FDA Perspective.

Why miltefosine-a life-saving drug for leishmaniasis-is unavailable to people who need it the most.

Miltefosine, the only oral drug approved for the treatment of leishmaniasis-a parasitic disease transmitted by sandflies-is considered as a success story of research and development (R&D) by a public-private partnership (PPP). It epitomises the multiple market failures faced by a neglected disease drug: patients with low ability to pay, neglect by authorities and uncertain market size. Originally developed as an anticancer agent in the 1990s, the drug was registered in India in 2002 to treat the fatal visce...

A comparison of the pharmacokinetic and pharmacodynamic properties of nitroglycerin according to the composition of the administration set: A preliminary study.

There is a risk of drug sorption into an intravenous administration set composed of polyvinyl chloride (PVC), polyurethane (PU), or polyolefin (PO). This has implications on the dose of the active ingredient the patient receives, and thus therapeutic success. This study aimed to determine the plasma concentration of nitroglycerin and the effect of nitroglycerin on patients based on the composition of the administration set.

Involvement of an orphan transporter, SLC22A18, in cell growth and drug resistance of human breast cancer MCF7 cells.

The SLC22A18 gene, which encodes an orphan transporter, is located at the 11p15.5 imprinted region, an important tumor-suppressor gene region. However, the role of SLC22A18 in tumor suppression remains unclear. Here, we investigated the involvement of SLC22A18 in cell growth, invasion and drug resistance of MCF7 human breast cancer cell line. Western blot analysis indicated that SLC22A18 is predominantly expressed at intracellular organelle membranes. Quantitative proteomics showed that knockdown of SLC22A1...

Natural history of 39 patients with Achondroplasia.

To characterize the natural history of 39 achondroplastic patients diagnosed by clinical, radiological and molecular assessments.

Structure-activity relationships of imidazothiazinones and analogs as antagonists of the cannabinoid-activated orphan G protein-coupled receptor GPR18.

GPR18 is a cannabinoid-activated orphan G protein-coupled receptor (GPCR) that is selectively expressed on immune cells. Despite its significant potential as a drug target for inflammatory diseases and cancer immunotherapy, only very few GPR18 ligands have been described to date. In the present study we investigated the structure-activity relationships (SARs) of (Z)-2-(3-(4-chlorobenzyloxy)benzylidene)-6,7-dihydro-2H-imidazo[2,1-b][1,3]thiazin-3(5H)-one (PSB-CB5, 5), the most potent GPR18 antagonist describ...


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